P783: CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN IN PATIENTS WITH PNH RECEIVING C5 INHIBITION TREATMENT ACROSS UK, GERMANY, AND FRANCE. INSIGHTS FROM THE COMMODORE BURDEN OF ILLNESS STUDY
Authors: Louis Terriou, Maria Piggin, Pascale Burmester, Fengkui Zhang, Alan Finnegan, Persefoni Kritikou, Claudia Mighiu, Christian Buehrer, Pablo Katz, Selma Mode, Xenia Studera, Huong Trinh, Talha Munir
Journal: HemaSphere (2023)

Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening blood disorder. In the UK, Germany, and France, C5 complement inhibitors, such as eculizumab and ravulizumab, both of which are intravenously (IV) infused, are the standard of care for PNH. Aims: The COMMODORE Burden of Illness study aims to quantify the real-life socioeconomic burden of PNH. We present results from the first study phase, for participants from the UK, Germany, and France, focusing on those receiving IV C5 inhibition treatment. Methods: Physicians completed Case Report Forms (CRFs) collecting information on clinical characteristics of the disease, at study enrollment. Following physician invitation, adult participants (≥18 years old) diagnosed with PNH ≥12 months prior to enrollment, and their caregivers, could complete patient-reported outcomes (PROs) and caregiver-reported outcome questionnaires, respectively. The clinical patient characteristics included PNH subtype and clinical events in the last 12 months. Patient questionnaires (PROs) included Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue), Patient Global Impression of Severity (PGI-S), Quality-of-Life Tool for Patients with Aplastic Anaemia and/or PNH (QLQ-AA/PNH-54), and 5-level EuroQoL 5-dimension (EQ-5D-5L). Caregiver questionnaires included the Carer Experience Scale (CES) and the Short Form 6 Dimension (SF-6Dv2.0). The economic burden was categorized as costs to the healthcare system (from the CRFs), costs to patients (from the Patient Questionnaires), and costs to caregivers (from the Caregiver Questionnaires). See table for cost types. Analyses were descriptive, and results are presented as n (%) or mean (range/standard deviation [SD]). Results: In total, 150 participants across all three countries treated with C5 inhibition were analyzed, comprising 150 CRFs, 58 Patient Questionnaires, and 33 Caregiver Questionnaires. The mean age at enrollment was 41.0 (range 20‒76) years, and the majority of participants were male (68.7%); 92.6% had classic PNH, and 7% had PNH with another bone marrow disease. Most participants received eculizumab as treatment (72.7%) vs ravulizumab (24.0%); while 3.3% switched from eculizumab to ravulizumab. 41.3% of the participants reported at least one thrombotic event and 50.7% experienced hemolysis in the prior 12 months. Patients (n=58) reported a mean FACIT-Fatigue score of 28.2 (SD 6.5); a mean PGI-S score of 4.5 (SD 2.3); for the QLQ-AA/PNH-54, the highest mean scores (indicating worse QoL) were associated with the “emotional functioning”, and the “cognitive functioning” components (69.3 [SD 16.9], and 68.1 [SD 17.4], respectively); and the mean EQ-5D-5L utility and visual analogue scale scores were 0.81 (SD 0.2), and 62.0 (SD 19.3), respectively. Caregivers (n=33) reported a mean CES score of 68.9 (SD 19.8) and a mean SF-6Dv2.0 utility score of 0.86 (SD 0.1). The mean annual total cost to the healthcare system per patient (N=150) was €350,122.7 (SD 118,508.1). Equivalently, the mean cost to patients (n=58) was €3,557.7 (SD 7,901.7), and the mean cost to the caregivers (n=33) was €1,740.4 (SD 3,788.7; n=33; table). Summary/Conclusion: The preliminary results presented here suggest that despite currently available IV C5 inhibition treatment, patients with PNH continue to experience substantial burden of disease, which translates into considerable costs and diminished QoL. Further, this is one of the first reports of outcomes from the PNH-specific QLQ-AA/PNH-54. The persistent unmet need and disease burden requires alternative treatment options and appropriate support.Keywords: Complement, Paroxysmal nocturnal hemoglobinuria (PNH), Patient reported outcomes, Real world data